Single subretinal injection of vector suspension; up to 3x10e12 vector particles

Other Name: rAAV 2/2.hRPE65p.hRPE65

Detailed Description:

The main objective of the proposed trial is to determine the safety and efficacy subretinal administration of a recombinant adeno-associated viral vector (rAAV 2/2.hRPE65p.hRPE65) at three different dosage levels in individuals with autosomal recessive severe early-onset retinal degeneration due to mutations in RPE65. We have a comprehensive clinical monitoring plan to investigate the safety and efficacy of vector delivery.

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Please refer to this study by its ClinicalTrials.gov identifier: NCT00643747